FDA Grants Priority Review to Huntington Disease Gene Therapy BLA, Sevasemten Misses Primary, Secondary End Points in BMD, Overviewing the 1-Year KYSA-8 Findings

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Welcome to the Neurology News Network, my name is Louie Pasculli and here’s a look at some of the top stories in Neurology.

Beginning with FDA news, the administration has accepted uniQure'sbiologics license application (BLA) for ifezuntirgeneinilparvovec(AMT-130), an investigational gene therapy for Huntington disease, and granted it Priority Review. The agency set a target action date of May 1, 2027, under the Prescription Drug User Fee Act (PDUFA), and said it is tentatively planning to hold an advisory committee meeting, though no date has been finalized.¹

The BLA was submitted under the accelerated approval pathway. Matt Kapusta, chief executive officer of uniQure, said the acceptance “marks an important milestone for the Huntington's disease community, which has waited generations for a treatment that changes the course of this devastating disease.” He added that the company, “backed by compelling data from our Phase I/II studies," believes ifezuntirgeneinilparvovec “has the potential to become the first disease modifying treatment for Huntington's disease.”¹

Ifezuntirgeneinil parvovec uses uniQure's gene-silencing miQURE platform to deliver a microRNA designed to silence the huntingtin gene and the toxic exon 1 protein fragment it produces. It is given as a single administration through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum. The therapy holds Breakthrough Therapy, RMAT (Regenerative Medicine Advanced Therapy), and Fast Track designations; it is the first Huntington disease candidate to hold both Breakthrough Therapy and RMAT status.

In other news, According to a recent news update from Servier, the company’s investigational agent sevasemten failed to meet its primary or secondary endpoints in the pivotal GRAND CANYON trial for Becker muscular dystrophy (BMD). The result reverses years of positive signals for a therapy that had been positioned to become the first approved treatment for the disease, which currently has none.²

GRAND CANYON is the pivotal cohort of a broader clinical program (NCT05291091) that also includes the phase 2 CANYON study. It randomized about 120 adults with BMD 2:1 to oral sevasemten or placebo over 18 months, with change in the North Star Ambulatory Assessment (NSAA) as the primary endpoint. Servier said sevasemten showed no statistically significant benefit on the NSAA and that unspecified secondary endpoints also were not met.

The company reported a continued favorable safety and tolerability profile and said it is conducting a comprehensive analysis of the GRAND CANYON dataset alongside prior clinical data, noting that the results are not aligned with findings from earlier studies, including CANYON. Full results are expected to be presented at a later date.

Switching gears, The phase 2 KYSA-8 study is evaluating a single infusion of miv-cel in adults with SPS who have had an inadequate response to immunomodulatory therapies. In its primary analysis, treatment demonstrated significant improvement in the timed 25-foot walk (T25FW) at week 16, alongside a favorable safety profile and immunotherapy-free remissions. Investigators have continued to follow participants to better understand the durability of these clinical responses and the potential effects of B-cell reconstitution following CAR T-cell therapy.

Updated 1-year findings from KYSA-8 were presented at the 10th Joint ACTRIMS-ECTRIMS Meeting (MSToronto 2026), held October 21-23, 2026, in Toronto, Ontario, Canada. To discuss the findings, NeurologyLive® spoke with Amanda L. Piquet, MD, professor of neurology at the University of Colorado Anschutz, who reviewed the sustained improvements observed across measures of walking ability, neurologic disability, and stiffness. Piquet also discussed the safety findings, including the absence of high-grade cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS), and the observation that clinical benefits continued despite B-cell reconstitution in a proportion of patients.

Looking ahead, Piquet emphasized the importance of longer-term follow-up to determine how durable these responses will be. Although early reports from compassionate-use cases have described sustained benefits beyond 2 years, continued monitoring of the KYSA-8 participants will help establish the long-term potential of CAR T-cell therapy in SPS.

To watch the full piece and to get more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to remain informed on the latest in neurology. I’m Louie Pasculli, thanks for watching Neurology News Network.

REFERENCES
1. uniQure announces FDA acceptance and priority review of the Biologics License Application for ifezuntirgene inilparvovec for the treatment of Huntington's disease. News release. uniQure. Published November 2, 2026. Accessed November 2, 2026. https://www.biospace.com/press-releases/uniqure-announces-fda-acceptance-and-priority-review-of-the-biologics-license-application-for-ifezuntirgene-inilparvovec-for-the-treatment-of-huntingtons-disease
2. Topline pivotal results of sevasemten in Becker muscular dystrophy. News release. Servier. Published November 3, 2026. Accessed November 5, 2026. https://servier.com/en/newsroom/topline-pivotal-results-sevasemten-becker/